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Exegenesis Bio, Modalis Partner on DMD Gene Therapy

The collaboration pairs Exegenesis Bio’s EMC181 muscle-tropic AAV capsid with Modalis’ CRISPR-GNDM platform to advance MDL-201.

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By: Charlie Sternberg

Associate Editor, Contract Pharma

Exegenesis Bio and Modalis Therapeutics Corporation have signed a research collaboration and license agreement to advance MDL-201, an innovative therapeutic candidate for Duchenne muscular dystrophy (DMD) based on Modalis’ proprietary CRISPR-GNDM (Guide Nucleotide-Directed Modulation) technology.

Under the agreement, Modalis will receive rights to use EMC181, an engineered muscle-tropic AAV capsid developed by Exegenesis Bio, for MDL-201. By combining Exegenesis Bio’s delivery technology with Modalis’ CRISPR-GNDM payload and development capabilities, the companies aim to accelerate research and development toward clinical application.

Zhenhua Wu, CEO of Exegenesis Bio, stated, “We are pleased to collaborate with Modalis to advance MDL-201. EMC181 was developed to enable efficient muscle targeting while reducing liver exposure, and we believe its combination with Modalis’ innovative CRISPR-GNDM payload represents a compelling approach for DMD. This collaboration reflects our strategy of applying our next-generation AAV capsid platform to differentiated gene therapies with the potential to address significant unmet medical needs.”

Haruhiko Morita, CEO of Modalis Therapeutics, said, “We are excited to partner with Exegenesis Bio to advance MDL-201. Combining our CRISPR-GNDM payload with EMC181 brings together two highly complementary technologies – precise, durable activation of utrophin and efficient muscle-targeted delivery. We believe this collaboration has the potential to accelerate MDL-201 toward the clinic and to establish a differentiated, mutation-agnostic treatment approach for DMD.”

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